EP3917522A4 - METHODS OF TREATMENT OF AAV VECTORS FOR TYPE 2 LATE INFANTILE NEURONAL CERIODIC LIPOFUSCINOSIS - Google Patents

METHODS OF TREATMENT OF AAV VECTORS FOR TYPE 2 LATE INFANTILE NEURONAL CERIODIC LIPOFUSCINOSIS Download PDF

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Publication number
EP3917522A4
EP3917522A4 EP20749051.7A EP20749051A EP3917522A4 EP 3917522 A4 EP3917522 A4 EP 3917522A4 EP 20749051 A EP20749051 A EP 20749051A EP 3917522 A4 EP3917522 A4 EP 3917522A4
Authority
EP
European Patent Office
Prior art keywords
lipofucinosis
treatment
methods
type
aav vector
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Withdrawn
Application number
EP20749051.7A
Other languages
German (de)
English (en)
French (fr)
Other versions
EP3917522A1 (en
Inventor
David William Anderson
Maryann TOTO
Sue E.I. DASEN
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Spark Therapeutics Inc
Original Assignee
Spark Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Spark Therapeutics Inc filed Critical Spark Therapeutics Inc
Publication of EP3917522A1 publication Critical patent/EP3917522A1/en
Publication of EP3917522A4 publication Critical patent/EP3917522A4/en
Withdrawn legal-status Critical Current

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Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/4813—Exopeptidases (3.4.11. to 3.4.19)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/14—Dipeptidyl-peptidases and tripeptidyl-peptidases (3.4.14)
    • C12Y304/14009—Tripeptidyl-peptidase I (3.4.14.9)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Immunology (AREA)
  • Veterinary Medicine (AREA)
  • Biotechnology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Transplantation (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicinal Preparation (AREA)
EP20749051.7A 2019-02-01 2020-01-31 METHODS OF TREATMENT OF AAV VECTORS FOR TYPE 2 LATE INFANTILE NEURONAL CERIODIC LIPOFUSCINOSIS Withdrawn EP3917522A4 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962800131P 2019-02-01 2019-02-01
PCT/US2020/016235 WO2020160486A1 (en) 2019-02-01 2020-01-31 Aav vector treatment methods for late infantile neuronal ceroid lipofuscinosis type 2

Publications (2)

Publication Number Publication Date
EP3917522A1 EP3917522A1 (en) 2021-12-08
EP3917522A4 true EP3917522A4 (en) 2022-09-28

Family

ID=71841678

Family Applications (1)

Application Number Title Priority Date Filing Date
EP20749051.7A Withdrawn EP3917522A4 (en) 2019-02-01 2020-01-31 METHODS OF TREATMENT OF AAV VECTORS FOR TYPE 2 LATE INFANTILE NEURONAL CERIODIC LIPOFUSCINOSIS

Country Status (9)

Country Link
US (1) US20220184188A1 (https=)
EP (1) EP3917522A4 (https=)
JP (2) JP2022518931A (https=)
CN (1) CN113891712A (https=)
AU (1) AU2020215592A1 (https=)
BR (1) BR112021015050A2 (https=)
CA (1) CA3127889A1 (https=)
MX (1) MX2021009250A (https=)
WO (1) WO2020160486A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3132379A1 (en) * 2019-03-22 2020-10-01 Rush University Medical Center Combination of nasal gene delivery and oral cinnamic acid, oleamide or gemfibrozil for lysosomal storage disorders
AU2022337146A1 (en) 2021-09-03 2024-03-14 Tacit Therapeutics, Inc. Rna editing via recruitment of spliceosome components
WO2025231406A1 (en) * 2024-05-02 2025-11-06 The Children's Hospital Of Philadelphia Methods to increase transduction of ependyma cells in brain

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3024497A2 (en) * 2013-07-26 2016-06-01 University of Iowa Research Foundation Methods and compositions for treating brain diseases

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2132309A4 (en) * 2007-02-23 2011-01-05 Univ Florida COMPOSITIONS AND METHODS OF TREATING GLYCOLOGICAL MEMORY DISEASES
WO2012135857A1 (en) * 2011-03-31 2012-10-04 University Of Iowa Research Foundation Methods and compositions for treating brain diseases
US20140271550A1 (en) * 2013-03-14 2014-09-18 The Trustees Of The University Of Pennsylvania Constructs and Methods for Delivering Molecules via Viral Vectors with Blunted Innate Immune Responses
US10900053B2 (en) * 2014-11-21 2021-01-26 University Of Florida Research Foundation, Incorporated Genome-modified recombinant adeno-associated virus vectors
KR102738860B1 (ko) * 2015-10-23 2024-12-04 유니버시티 오브 아이오와 리써치 파운데이션 리소좀 축적 질환 (lsd)을 갖는 포유동물의 치료 방법에 사용하기 위한 aav 입자를 포함하는 약제학적 조성물
CA3041548A1 (en) * 2016-11-04 2018-05-11 The Children's Hospital Of Philadelphia Gene transfer compositions, methods and uses for treating neurodegenerative diseases

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3024497A2 (en) * 2013-07-26 2016-06-01 University of Iowa Research Foundation Methods and compositions for treating brain diseases

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
DOLAN SONDHI ET AL: "Long-Term Expression and Safety of Administration of AAVrh.10hCLN2 to the Brain of Rats and Nonhuman Primates for the Treatment of Late Infantile Neuronal Ceroid Lipofuscinosis", HUMAN GENE THERAPY METHODS, vol. 23, no. 5, 1 October 2012 (2012-10-01), pages 324 - 335, XP055271964, ISSN: 1946-6536, DOI: 10.1089/hgtb.2012.120 *
See also references of WO2020160486A1 *

Also Published As

Publication number Publication date
JP2025098018A (ja) 2025-07-01
CN113891712A (zh) 2022-01-04
CA3127889A1 (en) 2020-08-06
JP2022518931A (ja) 2022-03-17
MX2021009250A (es) 2021-11-04
WO2020160486A1 (en) 2020-08-06
US20220184188A1 (en) 2022-06-16
AU2020215592A1 (en) 2021-08-12
BR112021015050A2 (pt) 2021-10-05
EP3917522A1 (en) 2021-12-08

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